1043-3155 Pediatr Neurol Briefs pedneurbriefs Pediatr Neurol Briefs Pediatric Neurology Briefs Pediatr Neurol Briefs 2166-6482 1043-3155 2166-3155 Pediatric Neurology Briefs Publishers Chicago, IL, USA PNB-7-62-a 10.15844/pedneurbriefs-7-8-7 Degenerative Diseases Neurology Pediatrics Nervous System Diseases Child Development Brain Diseases Neurosurgery Child Infant Lorenzo Oil Therapy for Adrenoleukodystrophy http://orcid.org/0000-0002-0173-7931 Millichap J. Gordon MD 1 2 * Division of Neurology, Children's Memorial Hospital, Chicago, IL Departments of Pediatrics and Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL Correspondence: Dr. J. Gordon Millichap, E-mail: jgmillichap@northwestern.edu 08 1993 01 07 2016 7 8 62 62 Copyright: © 1993 The Author(s) 1993 This work is licensed under the Creative Commons Attribution 4.0 International License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Visual evoked potentials in adrenoleukodystrophy: a trial with glycerol trioleate and Lorenzo oil

Dietary therapy with glycerol trioleate and glycerol trierucate (Lorenzo oil) was tested in 108 adult patients with adrenomyeloneuropathy phenotype of adrenoleukodystrophy (ALD) at Johns Hopkins Hospital and the Kennedy Krieger Institute, Baltimore, MD.

 Lorenzo Oil Adrenoleukodystrophy Very-Long-Chain Fatty Acid

Dietary therapy with glycerol trioleate and glycerol trierucate (Lorenzo oil) was tested in 108 adult patients with adrenomyeloneuropathy phenotype of adrenoleukodystrophy (ALD) at Johns Hopkins Hospital and the Kennedy Krieger Institute, Baltimore, MD. Pattern-reversal visual evoked potentials were used to evaluate visual pathways before and after treatment for 1 year. Very-long-chain fatty acid (VLCFA) levels were markedly reduced, but visual evoked potentials remained abnormal or became abnormal. No patients improved, and there was no evidence that reduction in VLCFA levels benefited or retarded demyelination of visual pathways. [1]

COMMENT. Moser, in an editorial [2], reviews the development of therapies for adrenoleukodystrophy and describes Lorenzo oil as “a prematurely amplified hope.” Trials of the oil in Europe and the USA have failed to demonstrate a significant effect on the rate of progression of the childhood cerebral form of ALD. A possible preventive effect of Lorenzo oil in patients who have not yet developed the neurological disability is under investigation. Of 61 asymptomatic patients treated for a few months up to 4 years at the Kennedy Krieger Institute, one has developed childhood cerebral ALD and 7 have shown progressive demyelination on the MRI. Longer follow-up is required to determine the outcome in the 53 who remain asymptomatic. A depression in platelets was an unexpected side effect of Lorenzo oil therapy.

 Kaplan PW Tusa RJ Shankroff J Heller J Moser HW Visual evoked potentials in adrenoleukodystrophy: a trial with glycerol trioleate and Lorenzo oil Ann Neurol 1993 Aug 34 2 169 174 10.1002/ana.410340212 8338341 Moser HW Lorenzo oil therapy for adrenoleukodystrophy: a prematurely amplified hope Ann Neurol 1993 Aug 34 2 121 122 10.1002/ana.410340202 8338333